HG-004

HG202 is the first CRISPR/Cas13Y RNA-targeting therapy to enter clinical development and the only clinical-stage CRISPR RNA-editing therapy for age-related macular degeneration Preclinical studies in laser-induced CNV mice showed HG202 reduced CNV area by 87%, outperforming both anti-VEGF antibodies and AAV-anti-VEGF gene therapy SHANGHAI and CLINTON, N.J., Nov. 4, 2024 /PRNewswire/ -- HuidaGene Therapeutics ("HuidaGene"), a global clinical-stage biotechnology company, announced that the U.S. FDA has cleared its investigational new drug (IND) application for HG202, the world's first-ever CRISPR/Cas13 RNA-editing therapy for clinical use in treating neovascular age-related macular degeneration (nAMD), which affects millions worldwide. "This open IND for HG202 by the U.S. FDA-the first regulator to clear CRISPR/Cas13 for clinical development–marks a significant milestone for HuidaGene and the CRISPR RNA-editing field," noted Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and CEO of HuidaGene. "The strong preclinical data for HG202, combined with promising results from the first-in-human ' SIGHT-I' trial in China, underscored its potential to address AMD using a non-receptor binding pathway through the Cas13 RNA editor to knockdown VEGF-A mRNA." Xin Zhang, M.D., MSc., COO and CMO of HuidaGene, highlighted the urgency of new nAMD treatments: "Up to 46% of AMD patients respond poorly or develop resistance to anti-VEGF therapies. AMD patients deserve safe, effective treatment options. The BRIGHT trial will evaluate HG202's safety and efficacy to address this gap. We look forward to enrolling patients soon." "Our AI/ML-driven HG-PRECISE® platform led us to discover the Cas13X/Y system (Nature Methods 2021)," added Hui Yang, Ph.D., Co-Founder and Chief Scientific Advisor of HuidaGene. "Building on this basis, my team engineered high-fidelity Cas13Y with efficient editing and low off-target effects (Nature Biotechnology 2022), laying out the technical foundation for future clinical applications." BRIGHT Trial of HG202 (NCT06623279) The BRIGHT (an open-la Bel, dose-escalation study for CRISPR/Cas13- RNA target In G t Herapy for the Treatment of neovascular AMD) trial, a Phase 1, dose-finding study, will investigate the safety and efficacy of HG202 at different doses in nAMD patients. Primary endpoints include the safety and tolerability of HG202, with secondary endpoints assessing improvements in visual acuity, retinal thickness, and the need for anti-VEGF injections. About HuidaGene HuidaGene utilizes its proprietary CRISPR-based HG-PRECISE® platform to develop potentially curative genome medicines. The Company is advancing clinical programs, including trials of HG004 (granted ODD & RPDD by FDA) ' LIGHT' trial (NCT06088992) and Phase 1/2 international, master-protocol ' STAR' clinical trial (NCT05906953) for RPE65-associated retinal disease, HG202 RNA-editing therapy ' SIGHT-I' first-in-human trial (NCT06031727) and ' BRIGHT' Phase 1 clinical trial (NCT06623279) for nAMD, HG204 RNA-editing therapy (granted ODD & RPDD by FDA and ODD by EMA) ' HERO' trial (NCT06615206) for MECP2 duplication syndrome, and HG302 DNA-editing therapy (granted ODD & RPDD by FDA) first-in-human ' MUSCLE' trial (NCT06594094) for DMD. The preclinical programs include HG303 DNA-editing for ALS and CRISPR RNA-editing therapy for Alzheimer's. With an extensive intellectual property portfolio, HuidaGene is a leader in genome medicines for neurology and ophthalmology. Learn more at huidagene.com or on LinkedIn. SOURCE HuidaGene Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SHANGHAI and REDWOOD CITY, Calif. , May 29, 2024 /PRNewswire/ -- HuidaGene Therapeutics ("HuidaGene"), a global clinical-stage biotechnology company focused on advancing CRISPR-based genomic medicines, and Synthego Corporation ("Synthego"), a leading provider of innovative CRISPR solutions for developing cell and gene therapies, proudly announce a partnered licensing agreement for the high-fidelity Cas12 CRISPR nuclease (hfCas12Max). This partnership underscores the significant clinical utility of hfCas12Max, naturally complementing Synthego's focus on advanced GMP manufacturing capabilities. Under this licensing agreement and pursuant to undisclosed financial terms between HuidaGene and Synthego, HuidaGene grants Synthego manufacturing and commercialization rights for the hfCas12Max nuclease and optimized gRNA for research; HuidaGene also grants Synthego the right to sublicense the nuclease for therapeutic use. This partnership will streamline the development of CRISPR-based therapeutic applications, providing developers with access to highly precise, efficient, and next-generation genome editing tools. Xuan Yao, Ph.D., Co-founder and President of HuidaGene, emphasized the significance of this agreement, stating, "HuidaGene's expansive CRISPR intellectual property portfolio distinguishes it as a vanguard in genomic medicine, with a versatile pipeline targeting critical neurological and ophthalmological conditions. Together with Synthego, we are poised to significantly accelerate the advancement of CRISPR-based therapies and deliver life-altering genomic medicines to patients worldwide." Craig Christianson, CEO of Synthego, expressed enthusiasm about the partnership with HuidaGene on hfCas12Max, stating, "The combination of our advanced CRISPR GMP production capabilities and regulatory expertise with HuidaGene's next-generation nuclease technology is a crucial step toward advancing transformative cell and gene therapies. This partnership underscores Synthego's commitment to CRISPR-based therapeutics, bolstered by our focused investments in GMP manufacturing and comprehensive CRISPR solutions that support therapeutic development. The integration of hfCas12Max naturally aligns with our initiative to enhance the accessibility and efficiency of CRISPR tools." hfCas12Max, a novel CRISPR gene-editing system developed through HuidaGene's HG-PRECISE platform, stands out for its superior on-target editing efficiency and reduced off-target editing activity in mammalian cells. This novel CRISPR gene-editing system also offers the advantage of packaging into a single viral vector, an important requirement for many cell and gene therapies. The commercialization of hfCas12Max will significantly enhance the accessibility of this CRISPR gene-editing system, providing increased freedom-to-operate and facilitating the development of CRISPR-based cell and gene therapies. This partnership reinforces both companies' positions as catalysts for innovation, advancing their shared mission of transforming genomic-based cell and gene therapies and enabling unprecedented therapeutic outcomes. For partnership inquiries, please contact [email protected] and [email protected]. hfCas12Max is a registered trademark in China. About HuidaGene HuidaGene Therapeutics utilizes its proprietary CRISPR-based HG-PRECISE® platform to discover, engineer, and develop potentially curative genomic medicine. The Company is advancing clinical programs of HG004 in RPE65-associated inherited retinal disease (granted both ODD and RPDD), HG202 CRISPR RNA-editing in neovascular age-related macular degeneration, and the preclinical pipeline, including HG204 CRISPR RNA-editing in neurodevelopmental disease of MECP2 duplication syndrome (granted both ODD and RPDD by U.S. FDA and ODD by EMA), HG302 CRISPR DNA-editing for Duchenne muscular dystrophy (granted both ODD and RPDD), and HG303 CRISPR DNA-editing for Amyotrophic Lateral Sclerosis (ALS). The Company's extensive intellectual property portfolio positions it as a leader in unleashing the full potential of genome medicine. Learn more at huidagene.com or on LinkedIn. About Synthego Synthego is a pioneering provider of genome engineering solutions, offering a comprehensive suite of products and services to accelerate CRISPR-based cell and gene therapy development. With a commitment to empowering researchers and innovators, Synthego's cutting-edge CRISPR technologies and expertise drive progress from preclinical to clinical application. Learn more at Synthego.com or on LinkedIn. SOURCE HuidaGene Therapeutics